NEJM publishes Phase 1 results of CRISPR editing of the KLKB1 gene in hereditary angioedema

Intellia Therapeutics/Lonvo-z (NTLA-2002)

NEJM published Phase 1 results of NTLA-2002 in 10 people with hereditary angioedema given single doses (25, 50 or 75 mg). Kallikrein protein fell 67% to 95% depending on dose; monthly swelling attacks dropped 95% on average, and side effects were mild.

• NTLA-2002 (lonvo-z) targets KLKB1 gene, silencing kallikrein production that drives swelling attacks in HAE.
• Dose-dependent response: 25 mg = 67% kallikrein reduction; 75 mg = 95% reduction.
• Patients remained attack-free for 5.5 to 10.6 months after a single infusion; mild infusion reactions and fatigue reported.
• One-time CRISPR infusion contrasts with current HAE treatments requiring frequent drug administration.